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Sarepta - MIS51ON

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Deletion Mutations Amenable to Exon 51 Skipping

Hub Summary

This phase 3 study is designed to evaluate the safety and tolerability of two doses of eteplirsen. Part One will be investigating two doses with Part 2 comparing the most effective dose from Part 1 with a 30mg/kg dose of eteplirsen. The UK sites have not yet been finalised, we will provide an update once we have these details.

Study Number: NCT03992430

Description by Sarepta Therapeutics

This study will be comprised of 2 parts: Part 1 will be conducted to evaluate the safety and tolerability of two doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 8 patients; Part 2 will be conducted for the selection of a high dose (high dose level 1 vs high dose level 2) (dose finding phase), and its comparison with the 30 mg/kg dose of eteplirsen (dose comparison phase), in approximately 144 DMD patients with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

Primary Outcome Measures

  1. Part 1 and Part 2 (Dose Finding): Incidences of Adverse Events (AEs) [ Time Frame: Baseline up to Week 148 ]
  2. Part 2 (Dose Finding): Dystrophin Expression in Biopsied Muscle Tissue [ Time Frame: Up to Week 48 ]
  3. Part 2 (Dose Finding): Pharmacokinetic (PK) Plasma Concentration of Eteplirsen [ Time Frame: Multiple timepoints up to Week 48 ]
  4. Part 2 (Dose Finding): Tissue Concentration of Eteplirsen From Biopsied Muscle Tissue [ Time Frame: Up to Week 48 ]
  5. Part 2 (Dose Comparison): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline, Week 144 ]

Secondary Outcome Measures

  1. Part 2 (Dose Comparison): Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) [ Time Frame: Baseline up to Week 144 ]
  2. Part 2 (Dose Comparison): Change From Baseline in Time to Complete Walk/run, Stairs and Time to Rise [ Time Frame: Baseline up to Week 144 ]
  3. Part 2 (Dose Comparison): Annual Rate in Decline of Forced Vital Capacity Percent Predicted (FVC%p) [ Time Frame: Up to Week 144 ]
  4. Part 2 (Dose Comparison): Time to Loss of Ambulation (LOA) [ Time Frame: Baseline up to Week 144 ]
  5. Part 2 (Dose Comparison): Change From Baseline in Skeletal Muscle Dystrophin Expression [ Time Frame: Baseline and Week 48 ]
  6. Part 2 (Dose Comparison): Incidence of Adverse Events (AEs) [ Time Frame: Baseline up to Week 144 ]

Can I take part?

Inclusion Criteria

  • Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
  • Have achieved a mean 6-minute walk test (6MWT) distance of greater than equal to (>=) 300 and less than equal to (<=) 450 meters.
  • Have intact right and left biceps muscles or an alternative upper arm muscle group.
  • Have been on a stable dose or dose equivalent of oral corticosteroids for at least 24 weeks prior to randomization.
  • Have stable pulmonary function (forced vital capacity >= 50 percent (%) of predicted and no requirement for nocturnal ventilation).

Exclusion Criteria

  • Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
  • Current or previous treatment with gene therapy or any other experimental pharmacologic treatment for DMD; some exceptions apply.
  • Previous treatment with drisapersen, ezutromid, or domagrozumab in the last 24 weeks prior to study enrollment.
  • Major surgery within 3 months prior to randomization.
  • Presence of any other significant neuromuscular or genetic disease other than DMD.
  • Presence of other clinically significant illness.
  • Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than (<) 50% on the screening Echocardiogram or QTcF >= 450 millisecond based on the screening ECGs.

Other inclusion/exclusion criteria apply.

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Overall Trial Status
Not yet recruiting
UK Locations
Trial Sponsor
Sarepta Therapeutics
Mutation Specific
Mutation specific therapies, must be amenable to exon 51 skipping
Muscle Biopsy
Muscle Biopsy Required
Length Of Participation
144 weeks
Recruitment Target
Therapeutic Category
Exon Skipping

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